HRP20220992T3 - Rasprostranjena isporuka gena motornim neuronima pomoću periferne injekcije aav vektora - Google Patents

Rasprostranjena isporuka gena motornim neuronima pomoću periferne injekcije aav vektora

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Publication number
HRP20220992T3
HRP20220992T3 HRP20220992TT HRP20220992T HRP20220992T3 HR P20220992 T3 HRP20220992 T3 HR P20220992T3 HR P20220992T T HRP20220992T T HR P20220992TT HR P20220992 T HRP20220992 T HR P20220992T HR P20220992 T3 HRP20220992 T3 HR P20220992T3
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HR
Croatia
Prior art keywords
aav
aav vector
motor neurons
gene
vector
Prior art date
Application number
HRP20220992TT
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English (en)
Inventor
Martine Barkats
Original Assignee
Genethon
Centre National De La Recherche Scientifique
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=39186802&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=HRP20220992(T3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Genethon, Centre National De La Recherche Scientifique filed Critical Genethon
Publication of HRP20220992T3 publication Critical patent/HRP20220992T3/hr

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
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  • Hospice & Palliative Care (AREA)
  • Transplantation (AREA)

Claims (4)

1. AAV vektor za uporabu u postupku za liječenje poremećaja motornog neurona kod subjekta kojem je to potrebno, pri čemu se navedeni vektor daje perifernom primjenom navedenog AAV vektora navedenom subjektu, navedeno davanje uzrokuje infekciju motornih neurona i ekspresiju gena u motornim neuronima, i pri čemu; navedeni AAV vektor sadrži - ljudsku AAV9 kapsidu; i - AAV-2 izvedeni genom koji sadrži gen od interesa koji kodira terapeutski proizvod, navedeni gen od interesa je operativno povezan s promotorom specifičnim ili funkcionalnim u motornim neuronima; pri čemu gen kodira protein "preživljavanja motornog neurona" (SMN); i pri čemu je poremećaj motornog neurona poremećaj spinalne mišićne atrofije (SMA).
2. AAV vektor za uporabu prema zahtjevu 1, naznačen time što navedena periferna injekcija sadrži intraperitonealnu (i.p.), intramuskularnu (i.m.) ili intravenoznu (i.v.) injekciju, poželjno intravenoznu injekciju.
3. AAV vektor za uporabu prema zahtjevu 1 ili 2, naznačen time što AAV genom je AAV genom s defektnom replikacijom kojem nedostaju funkcionalne Rep i Cap kodirajuće virusne sekvence.
4. AAV vektor za uporabu prema bilo kojem od zahtjeva 1 do 3, naznačen time što je AAV dvolančani samo-komplementarni genom.
HRP20220992TT 2007-10-05 2008-10-03 Rasprostranjena isporuka gena motornim neuronima pomoću periferne injekcije aav vektora HRP20220992T3 (hr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP07301435A EP2058401A1 (en) 2007-10-05 2007-10-05 Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP19158152.9A EP3505635B1 (en) 2007-10-05 2008-10-03 Widespread gene delivery to motor neurons using peripheral injection of aav vectors

Publications (1)

Publication Number Publication Date
HRP20220992T3 true HRP20220992T3 (hr) 2022-11-11

Family

ID=39186802

Family Applications (2)

Application Number Title Priority Date Filing Date
HRP20220992TT HRP20220992T3 (hr) 2007-10-05 2008-10-03 Rasprostranjena isporuka gena motornim neuronima pomoću periferne injekcije aav vektora
HRP20140643AT HRP20140643T1 (hr) 2007-10-05 2014-07-07 Dalekosežno dostavljanje gena motoriäśkim neuronima koristeä†i periferno injiciranje aav vektora

Family Applications After (1)

Application Number Title Priority Date Filing Date
HRP20140643AT HRP20140643T1 (hr) 2007-10-05 2014-07-07 Dalekosežno dostavljanje gena motoriäśkim neuronima koristeä†i periferno injiciranje aav vektora

Country Status (18)

Country Link
US (3) US9926574B2 (hr)
EP (5) EP2058401A1 (hr)
JP (2) JP5620819B2 (hr)
CN (2) CN105087650A (hr)
CA (2) CA2999607A1 (hr)
CY (1) CY1125608T1 (hr)
DK (2) DK2212424T3 (hr)
ES (3) ES2924892T3 (hr)
FI (1) FI2212424T4 (hr)
FR (2) FR20C1057I2 (hr)
HR (2) HRP20220992T3 (hr)
HU (2) HUE059864T2 (hr)
LT (2) LT3505635T (hr)
NO (1) NO2022048I1 (hr)
PL (2) PL3505635T3 (hr)
PT (3) PT2212424E (hr)
SI (2) SI3505635T1 (hr)
WO (1) WO2009043936A1 (hr)

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